New drug for hypertrophic cardiomyopathy appears well tolerated and effective

21st July 2016

A drug being developed to treat the underlying cause of hypertrophic cardiomyopathy (HCM) appeared to be safe and effective in initial trials.  So phase 2 trials will now go ahead, says its maker MyoKardia.

The drug, MYK-461, is designed to reduce excessive heart muscle contraction.

The company announced the successful completion of phase 1 trials earlier this month.

It said that data from the early trials suggested that people with the disease and healthy volunteers showed favourable tolerability to single doses of the drug and repeat doses for 28 days, and the drug reduced excessive heart muscle contractility

“These data indicate that MYK-461 has the potential to treat patients with hypertrophic cardiomyopathy, said associate professor Euan Ashley from Stanford University Medical Centre in America and a researcher in the MYK-461 development programme.

He added: ““MYK-461, if approved, may be the first drug to target the underlying biomechanical cause of this debilitating disease.”

The company says that later this year it will initiate a phase 2 study focusing on obstructive hypertrophic cardiomyopathy, in which the thickened heart muscle obstructs the outflow of blood from the heart.

Earlier this year the drug won special support in America, being granted orphan drug designation by the Food and Drug Administration. This designation, which supports the development of medicines in underserved or rare disorders, gives the company various financial benefits.

At the time, chief executive Tassos Gianakakos said: “This represents another step toward filling a critical need for patients and families who struggle with a chronic and debilitating illness for which there are no approved therapies.”

HCM with obstruction can cause symptoms including shortness of breath and chest pain. Patients are typically prescribed one or more drugs (including beta-blockers, calcium channel blockers and disopyramide) indicated for the treatment of hypertension, heart failure or other cardiovascular disorders more generally. Those with more severe disease or more symptoms, may require surgery or other invasive treatments.

For more information about HCM, see here.